pediatric drug
TRANSCRIPT
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Pediatric Drug & Medical Device Development: Setting Specifications & Defining Expectations
Purpose, Vision and Expected Outcomes
February 26, 2009
Carole Marcus, MBBChCo-Chair
Pediatric Drugs and Devices SubcommitteeCTSA Consortium Child Health Oversight Committee
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Meeting Purpose
To formulate specifications for a national child health clinical research infrastructure to effectively and efficiently develop drugs and medical devices for children
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Lack of Pediatric Information• ~ 80% of listed medication labels
disclaimed usage or lacked dosing information for children
– Physician’s Desk Reference 1973 & 1991 surveys
• Only 20-30 % of drugs approved by the FDA are labeled for pediatric use
– 1984-1989* survey, 1991-2001 repeat survey
• Only 38% of new drugs potentially useful in pediatrics were labeled for children when initially approved
– *1991-1997, FDA statistics
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3Allergy/asthma
8Steroids
13Respiratory
13Antibiotics
13Sedation/anesthestics
17Immunologic/rheumatologic
18Metabolic
21Antifungals/antiparasitics
21Anticonvulsants
25Endocrine
30Antivirals
32Analgesics
36Psychiatric
38Neurology
40Hematology
44Derm/eye/ear
44Antidotes
54Gastroenterology
57Cardiac
Percent Off-LabelClass of Medications
Off-Label Status of Medication AvailableFor Pediatric Use, by Class
Need For Pediatric Drug Development
Yoon, Clin Pediatr 2006; 45:75
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Need For Pediatric Medical Device Development
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Pediatric Considerations
• Children are currently underserved by the medical/scientific community
• Clinical research requirements for children are different than those for adults
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What CTSA’s Bring To The Table
• Intellectual and academic input and experience• Patient resources (common and rare diseases)• Infrastructure for research:
– Clinical research space– Laboratory resources– Clinical core facilities
• Development of central core facilities and informatics
• Education and training opportunities
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Pediatrics In The CTSA
• 37 of 38 CTSA’s have a pediatric component• 34 of the 38 CTSA’s sent pediatric
representatives to the 2008 Child Health Oversight Committee meeting.
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Current Problems With Pediatric Academic Multicenter Studies
• IRB delays• Technology transfer delays• Scientific review delays
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Process for Opening a Clinical Trial
Dilts, J Clin Oncol 2006; 24:4545
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Dilts, J Clin Oncol 2006; 24:4545
Mean Number Of Days Per Process
0 50 100 150 200 250 300
Start to 1st process
IRB
Contracts & grants
Scientific review committees
Receipt to open
Open to 1st subject
Receipt to 1st subject
58
69
100
105
189
73
251
Mean time (days)
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Today’s Goals
• The expected deliverable will be information and specifications on the infrastructure features and parameters for a national pediatric clinical and translational platform.
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Workshop Expectations
• There is no expectation to achieve consensus or solve problems, but to provide guidance and information to help develop the most responsive infrastructure.
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What We Are Not Going to Do!
• Discuss specific drug/device needs• Discuss funding mechanisms• Prioritize areas of research• Discuss regulatory issues Separate meetings / activities on many of
the above are planned by NIH.
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Specific Topics For Discussion
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Legal And Financial
• Use of templates and common documents for intellectual property issues
• Development of policies for intellectual property, data sharing and publication
• Development of policies for confidential information sharing and disclosure
• Potential mechanisms for cost sharing• Privacy and protecting health information
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FDA and OHRP Regulatory Support
• Regulatory support and regulatory resources
• Regulatory reporting information flow and responsibilities
• Interest and ability to hold IND’s and IDE’s
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Availability And Development Of Core Facilities
• Analytic services:– Pharmacokinetics– Pharmacogenomics– Pharmacodynamics– toxicology
• Specimen analysis• Specimen repository• Certification of core facilities
– GLP– CLIA
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Pediatric-specific Biostatistical Resources
• Longitudinal analyses• Incorporation of growth parameters into
statistical assessments• Small sample sizes
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Informatics Needs
• Use of data coordination centers• Use of data standards for data
acquisition, data transmission and data storage, to ensure interoperability
• Data security procedures
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Pediatric-specific Ethical Issues
• Guidelines for what constitutes minimal risk; criteria for referring protocols for 407 review
• Use of harmonized age-appropriate assent templates
• Guidelines for re-consenting pediatric enrollees in long term studies
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Bridging the Gap From Clinical Care To Research
• Approaches to integrate participation in clinical trials with clinical care
• Tools to inform families about pediatric clinical research
• Recruitment and retention policies including inclusion of underrepresented populations
• Outreach efforts• Sharing of results with study participants and
the general public (e.g., clinicaltrials.gov)
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Performance Metrics
• Plans to develop, implement and assess performance metrics
• Time to opening of study / enrollment of 1st subject
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Logistical Considerations
• Need for a single point of contact for general information for network activities
• Need for public web site; which elements to include
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Building A Culture Of Product Development
• Encourage product development in addition to pursuit of non-product development scientific agendas
• Anticipated training needs for consortium personnel to address these needs
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What To Expect From NIH
• Willingness to learn and adapt• Commitment to the highest scientific and ethical
standards• Willingness to collaborate and cooperate with
partners, patients, study participants and the public
• Interest in participating in the development of products with clinical benefit for children
Steven Hirschfeld, MD, PhD